By Barry Sudbeck and Laura Musgrave
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Here’s a question more pharma executives are asking: does patient engagement deliver measurable value, or is it primarily reputational?
It’s a fair question.
In an era where pharmaceutical innovation must demonstrate its value not only through clinical efficacy but also through demonstrated patient relevance, the question is no longer ‘whether’ to engage patients – it’s whether that engagement translates into an advantage.
New research from FleishmanHillard’s Global Health & Life Sciences group suggests it may. Released in recognition of Rare Disease Day, The Patient Engagement Premium: Defining the Strategic Value of Patient Input in Drug Development analyses FDA submissions for rare disease therapies approved between 2018 and 2024 and finds directional associations between documented patient input and regulatory outcomes.
From Philosophy to Evidence
The shift from transactional patient engagement to embedded patient evidence isn’t new thinking though, but it is accelerating in practice. As regulatory scrutiny of traditional DTC channels intensifies and Health Technology Assessment bodies increasingly consult patient advocacy organisations, companies face a choice: embed patient evidence directly into development processes, or risk losing ground to those who do.
Executive decision-makers however, demand more than anecdote. This research represents a crucial step towards establishing a measurable evidence base for patient engagement as a strategic investment, rather than solely a statement of values.
A Rigorous Approach to a Complex Question
The analysis examined 179 rare disease medicine approvals that included Patient Experience Data (PED) tables, a requirement formalised following the 21st Century Cures Act. Each product was assigned a ‘Patient Engagement Score’ based on six distinct engagement activities, ranging from patient advisory committee insights to patient-reported outcomes (PROs) and clinical outcome assessments (COAs).
Key findings include:
- Patient input is increasingly embedded in regulatory submissions. Nearly nine in ten submissions in 2023-2024 explicitly cited at least one patient engagement activity, representing a marked increase compared with earlier in the study period. PRO and COA data have become the most common forms of patient input, suggesting companies may be integrating patient insights more systematically and earlier in development.
- Higher engagement scores trended with patient-centred labelling. Products with label claims linked to patient input averaged 1.4 documented engagement categories, compared with 1.0 for those without, a modest but directional association that may offer commercial advantage.
- Company size isn’t a barrier. Mid-cap sponsors engaged in patient-centred activities almost as frequently as larger pharmaceutical companies, indicating that the potential benefits of patient engagement appear accessible across the competitive landscape.
What Happens to Companies That Don’t Move?
Let’s be clear: the evidence base is still developing, and these associations are directional rather than conclusive. But the implications are hard to ignore.
Patient engagement is evolving from an ethical consideration to a strategic necessity. Companies are prioritising structured, quantifiable patient data, particularly PROs and COAs, for FDA submissions. Yet many underutilise other pathways, including patient organisation partnerships and patient preference studies. This suggests that comprehensive investment in the full spectrum of patient evidence could represent an untapped competitive edge.
For smaller companies not yet systematically integrating patient perspectives, the takeaway is encouraging, structured engagement may level the playing field. For larger companies that under-invest in patient input, the risk is equally clear, patient-centred rivals may be building advantages that compound over time.
Looking Ahead
As the evidence base expands and sponsors document patient engagement more comprehensively, clearer patterns will likely emerge. However, the direction of travel is already evident: regulators, payers, and patients themselves are reshaping how innovation is valued. Companies that embed patient engagement as foundational rather than peripheral, will compound advantage across regulatory, payer and reputational landscapes. The infrastructure to do this exists. The question lies in the execution.
Our approach combines regulatory expertise with data science and AI tools to help clients operationalise patient input across the product lifecycle, ensuring innovation is positioned as both evidence-driven and human-centred.
The pharmaceutical industry is at an inflection point. The companies that treat patient engagement as foundational – not peripheral – will define what comes next.
To access the full report or discuss how strategic patient engagement can create value for your organisation, contact Barry Sudbeck and Laura Musgrave, Patient Engagement Specialists with FleishmanHillard’s Global Health & Life Sciences group.
